Pediatric Stem Cell Transplantation
Hematopoietic Stem Cell Transplant Clinic Information
Lucile Packard Children's Hospital
725 Welch Road
Palo Alto, CA 94304
Phone: (650) 498-7836
Fax: (650) 724-1164
Mondays, Wednesdays, Thursdays,
8:30 am-5:00 pm
With the creation of the Stanford Institute for Cancer/Stem Cell Biology and Medicine in 2002, Stanford has taken the lead in the global effort to unlock the regenerative powers of stem cells, precursor cells in the human body with the ability to divide continually and differentiate into specialized cell types.
The program in Stem Cell Transplantation works to translate today's ground-breaking discoveries into state-of–the-art care for a number of devastating hematological disorders, immunodeficiency disorders and cancers impacting children around the world.
By understanding how stem cells work and the signals that drive their differentiation, Stanford researchers envision one day devising patient-specific therapies based on his or her own stem cells. These cells could be used to replenish tissues or specialized cells damaged by cancer and other diseases or to grow genetically identical replacements for organs damaged by trauma.
Diseases that can be treated with hematopoietic stem cell transplantation
- Malignancies such as acute or chronic leukemia, Hodgkin's lymphoma or non-Hodgkin's lymphoma
- Selected solid tumors such as neuroblastoma and rhabdomyosarcoma
- Hematological disorders such as:
- Severe aplastic anemia
- Fanconi anemia
- Thalassemia
- Selected cases of sickle cell anemia
- Immunodeficiency disorders including severe combined immunodeficiency and Wiskott-Aldrich syndrome
- Selected inborn errors of metabolism (storage diseases)
Rajni Agarwal-Hashmi, MD
Clincal Director
SCT Service at LPCH
Areas of active research
Non-Myeloablative Transplants
Our team is participating in some of the early clinical trials for a promising new treatment called non-myleoablative hematopoietic stem cell transplants.
Although researchers and clinicians have made much progress in improving the outcomes of hematopoietic stem cell transplants, the risk of developing complications after an allogeneic transplant (a transplant using bone marrow or peripheral blood stem cells provided by a donor) is still substantial.
The goal of a non-myeloablative allogeneic hematopoietic stem cell transplantation is to offer the benefit of a standard transplant, but with reduced toxicity. In a standard transplant procedure, a child undergoes very high dose chemotherapy -- with or without radiation therapy -- to destroy as many cancer cells as possible before the healthy donor stem cells are transplanted into the patient.
Because the treatment also destroys the patient's bone marrow and immune system, the donor's stem cells are not rejected, and grow to replace the patient's bone marrow. A non-myeloablative transplant uses much smaller and less toxic doses of chemotherapy and radiation, allowing the stem cells of both the patient and the donor to coexist (a condition known as immune tolerance). This partial replacement of the bone marrow function can cure many diseases while reducing the severity of treatment side effects.
Relapse After Transplant
Relapse remains a significant threat following a bone marrow transplant for leukemia. Our physicians are looking for alternative treatment approaches to further reduce the risk of relapse. One option they are researching is the use of leukocyte, or white blood cell, infusions to encourage the new bone marrow to graft and then attack the leukemia cells. Another study is investigating the use of a novel pre-transplant therapy prior to the transplant, which will attack and weaken the leukemia cells.
Our doctors are also developing new ways to process bone marrow in the laboratory to make it more acceptable to the recipient. This will allow doctors to broaden the availability of appropriate donors.
Graft-Versus-Host Disease
Graft-versus-host disease (GVHD) is a condition that occurs when the newly regenerated immune system arising from an allogeneic transplant attacks the patient's body and damages organs and tissues. The drugs that a recipient of an allogeneic transplant takes to suppress their immune system and reduce the chance of developing GVHD also make them susceptible to developing a serious infection. Lucile Packard Children's Hospital physicians are working on new drugs to diminish the risk of GVHD following hematopoietic stem cell transplant while minimizing the risk of infection.
